CAMBRIDGE, Mass. & PARIS--(EON: Enhanced Online News)--Alnylam
Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics
company, and Sanofi
announced today a strategic restructuring of their RNAi therapeutics
alliance to streamline and optimize development and commercialization of
certain products for the treatment of rare genetic diseases.
Specifically, Alnylam will obtain global development and
commercialization rights to its investigational RNAi therapeutics
programs for the treatment of ATTR amyloidosis, including patisiran and
ALN-TTRsc02. Sanofi will receive royalties based on net sales of these
ATTR amyloidosis products. Sanofi will obtain global development and
commercialization rights to fitusiran, an investigational RNAi
therapeutic, currently in development for the treatment of people with
hemophilia A and B. Global commercialization of fitusiran, upon
approval, will be done by Sanofi Genzyme, the specialty care global
business unit of Sanofi. Alnylam will receive royalties based on net
sales of fitusiran products. With respect to other products falling
under the RNAi therapeutics alliance, the material terms of the 2014
Alnylam-Sanofi Genzyme alliance remain unchanged.

“This strategic restructuring enables streamlined development and an
optimized approach to bringing innovative medicines to patients with
ATTR amyloidosis and hemophilia around the world, maximizing the
commercial opportunities for these programs,” said John Maraganore,
Ph.D., Chief Executive Officer of Alnylam. “For Alnylam, this provides
strategic clarity and operational alignment with regard to the
development and commercialization of patisiran and ALN-TTRsc02. This
will allow us to develop both products in a comprehensive manner,
potentially addressing the full spectrum of transthyretin-mediated
amyloidosis disease treatment and prevention. At the same time, we will
continue to support and benefit – via royalties – from the fitusiran
opportunity through Sanofi’s significant development and commercial
leadership.”

This restructuring provides Alnylam with the opportunity to consolidate
its ATTR amyloidosis business to maximize its value, and the opportunity
for near-term acceleration of product revenue growth based on newly
obtained rights to commercialize patisiran around the world, once
approved. In addition, it enables Alnylam to build a global presence and
commercial infrastructure that can be leveraged for ALN-TTRsc02 and
additional programs – including givosiran, an investigational RNAi
therapeutic for the treatment of acute hepatic porphyrias, and
cemdisiran, an investigational RNAi therapeutic for the treatment of
complement-mediated diseases – where Alnylam has retained global
ownership.

“The restructured alliance reflects Sanofi Genzyme’s sustained interest
in the strong potential of Alnylam’s portfolio of genetic medicines. The
new structure simplifies operations, providing both parties the agility
needed to make these medicines available to patients as quickly as
possible once approved,” said Bill Sibold, Executive Vice President and
Head of Sanofi Genzyme. “This restructuring will enable both parties to
maximize the value of each asset and allows us to maintain shared
economics across the alliance program.”

Fitusiran complements Sanofi Genzyme’s rare hematology portfolio, and
creates a focus on bringing an innovative product to market globally,
upon approval, for people living with hemophilia, one of the most common
rare diseases.

Terms of the Agreements

Patisiran and ALN-TTRsc02

Alnylam will fund all development and commercialization costs for
patisiran and ALN-TTRsc02 going forward. There will be no additional
milestones due to either company with respect to patisiran or
ALN-TTRsc02. Sanofi intends to substantially complete the transition of
its patisiran activities in regions outside the United States, Canada,
and Western Europe, consistent with the original scope of its license
rights to patisiran, by mid-2018.

Fitusiran

The restructuring will enable Sanofi to assume full responsibility for
development and commercialization of fitusiran, including costs.
However, during the anticipated transition period Alnylam will fund such
costs. Alnylam intends to substantially complete the transition of
fitusiran to Sanofi by mid-2018. Sanofi will pay Alnylam a milestone of
$50 million following dosing of the first patient in the ATLAS Phase 3
program for fitusiran.

Product Royalties

Alnylam and Sanofi Genzyme will be eligible to receive tiered royalties
of 15 to 30 percent on global net sales of fitusiran and ALN-TTRsc02,
respectively, upon approval and commercialization. Previously, these
programs were subject to co-development and co-commercialization terms
in the United States, Canada, and Western Europe. For patisiran, Sanofi
Genzyme will be eligible to receive royalties, increasing over time to
up to 25 percent, on sales in territories excluding the United States,
Canada, and Western Europe. Sanofi continues to have the right to opt
into other Alnylam rare genetic disease programs for development and
commercialization in territories outside of the United States, Canada,
and Western Europe, as well as one right to a global license.

The transaction is subject to customary closing conditions and
clearances, including clearance under the Hart-Scott Rodino Antitrust
Improvements Act.

Conference Call Details

Alnylam management will discuss this restructured alliance in a
conference call on January 7, 2018 at 4:30 p.m. ET, 1:30 p.m. PT. A
slide presentation will also be available on the Investors page of the
Company's website, www.alnylam.com,
to accompany the conference call. To access the call, please dial (877)
312-7507 (domestic) or (631) 813-4828 (international) five minutes prior
to the start time and refer to conference ID 3786228. A replay of the
call will be available beginning at 7:30 p.m. ET, 4:30 p.m.
PT on January 7, 2018. To access the replay, please dial (855) 859-2056
(domestic) or (404) 537-3406 (international), and refer to conference ID
3786228.

About RNAi

RNAi (RNA interference) is a natural cellular process of gene silencing
that represents one of the most promising and rapidly advancing
frontiers in biology and drug development today. Its discovery has been
heralded as “a major scientific breakthrough that happens once every
decade or so,” and was recognized with the award of the 2006 Nobel Prize
for Physiology or Medicine. By harnessing the natural biological process
of RNAi occurring in our cells, a major new class of medicines, known as
RNAi therapeutics, is on the horizon. Small interfering RNA (siRNA), the
molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic
platform, function upstream of today’s medicines by potently silencing
messenger RNA (mRNA) – the genetic precursors – that encode for
disease-causing proteins, thus preventing them from being made. This is
a revolutionary approach with the potential to transform the care of
patients with genetic and other diseases.

About Alnylam Pharmaceuticals

Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference
(RNAi) into a whole new class of innovative medicines with the potential
to transform the lives of people afflicted with rare genetic,
cardio-metabolic, and hepatic infectious diseases. Based on Nobel
Prize-winning science, RNAi therapeutics represent a powerful,
clinically validated approach for the treatment of a wide range of
severe and debilitating diseases. Founded in 2002, Alnylam is delivering
on a bold vision to turn scientific possibility into reality, with a
robust discovery platform and deep pipeline of investigational
medicines, including four product candidates that are in late-stage
development. Looking forward, Alnylam will continue to execute on its
"Alnylam 2020" strategy of building a multi-product, commercial-stage
biopharmaceutical company with a sustainable pipeline of RNAi-based
medicines to address the needs of patients who have limited or
inadequate treatment options. Alnylam employs over 700 people in the
U.S. and Europe and is headquartered in Cambridge, MA. For more
information about our people, science and pipeline, please visit www.alnylam.com
and engage with us on Twitter at @Alnylam
or on LinkedIn.

About Sanofi

Sanofi is dedicated to supporting people through their health
challenges. We are a global biopharmaceutical company focused on human
health. We prevent illness with vaccines, provide innovative treatments
to fight pain and ease suffering. We stand by the few who suffer from
rare diseases and the millions with long-term chronic conditions.

With more than 100,000 people in 100 countries, Sanofi is transforming
scientific innovation into healthcare solutions around the globe.

Sanofi Genzyme focuses on developing specialty treatments for
debilitating diseases that are often difficult to diagnose and treat,
providing hope to patients and their families. Learn more at www.sanofigenzyme.com.

Sanofi, Empowering Life.

Alnylam Forward Looking Statements

Various statements in this release concerning Alnylam’s future
expectations, plans and prospects, including without limitation,
Alnylam's views with respect to the potential for streamlined
development and an optimized approach to bringing innovative medicines
to patients with ATTR amyloidosis and hemophilia around the world,
maximizing the commercial opportunities for these programs under the
restructured alliance with Sanofi Genzyme, the development of patisiran
and ALN-TTRsc02 in a comprehensive manner, addressing the full spectrum
of transthyretin-mediated amyloidosis disease treatment and prevention,
expectations regarding a potential milestone payment and potential
royalty payments under the restructured alliance, and expectations
regarding its "Alnylam 2020" guidance for the advancement and
commercialization of RNAi therapeutics, constitute forward-looking
statements for the purposes of the safe harbor provisions under The
Private Securities Litigation Reform Act of 1995. Actual results and
future plans may differ materially from those indicated by these
forward-looking statements as a result of various important risks,
uncertainties and other factors, including, without limitation,
Alnylam's ability to discover and develop novel drug candidates and
delivery approaches, successfully demonstrate the efficacy and safety of
its product candidates, the pre-clinical and clinical results for its
product candidates, which may not be replicated or continue to occur in
other subjects or in additional studies or otherwise support further
development of product candidates for a specified indication or at all,
actions or advice of regulatory agencies, which may affect the design,
initiation, timing, continuation and/or progress of clinical trials or
result in the need for additional pre-clinical and/or clinical testing,
delays, interruptions or failures in the manufacture and supply of its
product candidates, obtaining, maintaining and protecting intellectual
property, Alnylam's ability to enforce its intellectual property rights
against third parties and defend its patent portfolio against challenges
from third parties, obtaining and maintaining regulatory approval,
pricing and reimbursement for products, progress in establishing a
commercial and ex-United States infrastructure, competition from others
using technology similar to Alnylam's and others developing products for
similar uses, Alnylam's ability to manage its growth and operating
expenses, obtain additional funding to support its business activities,
and establish and maintain strategic business alliances and new business
initiatives, Alnylam's dependence on third parties for development,
manufacture and distribution of products, the outcome of litigation, the
risk of government investigations, and unexpected expenditures, as well
as those risks more fully discussed in the "Risk Factors" filed with
Alnylam's most recent Quarterly Report on Form 10-Q filed with
the Securities and Exchange Commission (SEC) and in other filings that
Alnylam makes with the SEC. In addition, any forward-looking statements
represent Alnylam's views only as of today, and should not be relied
upon as representing its views as of any subsequent date. Alnylam
explicitly disclaims any obligation, except to the extent required by
law, to update any forward-looking statements.

Sanofi Forward Looking Statements

This press release contains forward-looking statements as defined in the
Private Securities Litigation Reform Act of 1995, as amended.
Forward-looking statements are statements that are not historical facts.
These statements include projections and estimates and their underlying
assumptions, statements regarding plans, objectives, intentions and
expectations with respect to future financial results, events,
operations, services, product development and potential, and statements
regarding future performance. Forward-looking statements are generally
identified by the words "expects", "anticipates", "believes", "intends",
"estimates", "plans" and similar expressions. Although Sanofi's
management believes that the expectations reflected in such
forward-looking statements are reasonable, investors are cautioned that
forward-looking information and statements are subject to various risks
and uncertainties, many of which are difficult to predict and generally
beyond the control of Sanofi, that could cause actual results and
developments to differ materially from those expressed in, or implied or
projected by, the forward-looking information and statements. These
risks and uncertainties include among other things, the uncertainties
inherent in research and development, future clinical data and analysis,
including post marketing, decisions by regulatory authorities, such as
the FDA or the EMA, regarding whether and when to approve any drug,
device or biological application that may be filed for any such product
candidates as well as their decisions regarding labelling and other
matters that could affect the availability or commercial potential of
such product candidates, the absence of guarantee that the product
candidates if approved will be commercially successful, the future
approval and commercial success of therapeutic alternatives, Sanofi's
ability to benefit from external growth opportunities and/or obtain
regulatory clearances, risks associated with intellectual property and
any related pending or future litigation and the ultimate outcome of
such litigation, trends in exchange rates and prevailing interest rates,
volatile economic conditions, the impact of cost containment initiatives
and subsequent changes thereto, the average number of shares outstanding
as well as those discussed or identified in the public filings with the
SEC and the AMF made by Sanofi, including those listed under "Risk
Factors" and "Cautionary Statement Regarding Forward-Looking Statements"
in Sanofi's annual report on Form 20-F for the year ended December 31,
2016. Other than as required by applicable law, Sanofi does not
undertake any obligation to update or revise any forward-looking
information or statements.

CAMBRIDGE, Mass.--(EON: Enhanced Online News)--For many people living with a rare disease, such as hereditary ATTR (hATTR) amyloidosis, it can take years to be accurately diagnosed. Moreover, many ... more »